Aeterna Zentaris Announces EMA Pediatric Committee Agreement on the Pediatric Investigation Plan for Macrilen™
March 07 2017 - 8:30AM
Business Wire
Acceptance of the Pediatric Investigation Plan paves the way for
the submission of a Marketing Authorization Application in Europe
for Macrilen™
Aeterna Zentaris Inc. (NASDAQ: AEZS)(TSX: AEZS) (the
“Company”) announced today that the Pediatric Committee (“PDCO”) of
the European Medicines Agency (“EMA”) agreed to the Company’s
Pediatric Investigation Plan (“PIP”) for Macrilen™ and agreed that
the Company may defer conducting the PIP until after it files a
Marketing Authorization Application (“MAA”) seeking marketing
authorization for the use of Macrilen™ for the evaluation of adult
growth hormone deficiency.
As part of the regulatory process for the registration of new
medicines in Europe, pharmaceutical companies are required to
provide a PIP outlining their strategy for investigation of the new
medicinal product in the pediatric population. An accepted PIP is a
prerequisite for filing an MAA for any new medicinal product in
Europe. However, the Company will be able to file an MAA
substantially earlier than if it was required to complete the PIP
before filing, because the PDCO permitted the Company to defer
conducting the studies defined in the PIP.
The Company’s PIP provides for a dose-escalation safety and
PK/PD study followed by a diagnostic efficacy and safety study. The
goal of the first study is to establish a dose that can safely be
administered to pediatric patients and that causes a clear rise in
growth hormone concentration in subjects ultimately diagnosed as
not having growth hormone deficiency (“GHD”). The recommended dose
derived from this study will be evaluated in the second study for
diagnostic efficacy and safety. Both studies will be conducted in
patients at least three years old who are undergoing the study
sites’ regular diagnostic evaluation for the presence of GHD.
Dr. Richard Sachse, M.D., Ph.D., Senior Vice President, Chief
Medical Officer and Chief Scientific Officer of the Company, said:
“PDCO’s agreement that we may defer conducting our PIP, allowing us
to go forward with an MAA in adults prior to initiating of the
pediatric development program, is an important step forward in our
efforts to bring Macrilen™ to market. Likewise, acceptance of our
PIP is an important regulatory milestone for the product. At this
time, we are looking forward to our upcoming FDA meeting, which has
been scheduled for late March 2017, as a pre-requisite for a
potential NDA submission based on our most recently announced
completion of the pivotal Phase 3 study. Similarly, we are
preparing for meetings with European agencies to discuss whether
the conducted development program suffices for an MAA.”
About MacrilenTM (macimorelin)
Macimorelin, a ghrelin receptor agonist, is an orally-active
small molecule that stimulates the secretion of growth hormone.
Macimorelin has been granted orphan drug designation by the FDA for
diagnosis of AGHD. The Company owns the worldwide rights to this
patented compound and has significant patent protection left. The
Company’s U.S. composition of matter patent expires in 2022 and its
U.S. utility patent runs through 2027. The Company proposes,
subject to FDA approval, to market macimorelin in the United States
under the tradename Macrilen™.
About AGHD
AGHD affects approximately 75,000 adults across the US, Canada
and Europe. Growth hormone not only plays an important role in
growth from childhood to adulthood, but also helps promote a
hormonally-balanced health status. AGHD mostly results from damage
to the pituitary gland. It is usually characterized by a reduction
in bone mineral density, lean body mass, exercise capacity, and
overall quality of life as well as an increase of cardiovascular
risks.
About Aeterna Zentaris Inc.
Aeterna Zentaris is a specialty biopharmaceutical company
engaged in developing and commercializing novel treatments in
oncology, endocrinology and women’s health. We are engaged in drug
development activities and in the promotion of products for others.
We recently completed Phase 3 studies of two internally developed
compounds. The focus of our business development efforts is the
acquisition of licenses to products that are relevant to our
therapeutic areas of focus. We also intend to license out certain
commercial rights of internally developed products to licensees in
non-US territories where such out-licensing would enable us to
ensure development, registration and launch of our product
candidates. Our goal is to become a growth-oriented specialty
biopharmaceutical company by pursuing successful development and
commercialization of our product portfolio, achieving successful
commercial presence and growth, while consistently delivering value
to our shareholders, employees and the medical providers and
patients who will benefit from our products. For more information,
visit www.aezsinc.com.
Forward-Looking Statements
This press release contains forward-looking statements made
pursuant to the safe harbor provision of the U.S. Securities
Litigation Reform Act of 1995, which reflect our current
expectations regarding future events. Forward-looking statements
may include, but are not limited to statements preceded by,
followed by, or that include the words “expects,” “believes,”
“intends,” “anticipates,” and similar terms that relate to future
events, performance, or our results. Forward-looking statements
involve known risks and uncertainties, many of which are discussed
under the caption “Key Information – Risk Factors” in our most
recent Annual Report on Form 20-F filed with the relevant Canadian
securities regulatory authorities in lieu of an annual information
form and with the U.S. Securities and Exchange Commission (“SEC”).
Such statements include, but are not limited to, statements about
the progress of our research, development and clinical trials and
the timing of, and prospects for, regulatory approval and
commercialization of our product candidates, the timing of expected
results of our studies, anticipated results of these studies,
statements about the status of our efforts to establish a
commercial operation and to obtain the right to promote or sell
products that we did not develop and estimates regarding our
capital requirements and our needs for, and our ability to obtain,
additional financing. Known and unknown risks and uncertainties
could cause our actual results to differ materially from those in
forward-looking statements. Such risks and uncertainties include,
among others, the availability of funds and resources to pursue our
research and development projects and clinical trials, the
successful and timely completion of clinical studies, the risk that
safety and efficacy data from any of our Phase 3 trials may not
coincide with the data analyses from previously reported Phase 1
and/or Phase 2 clinical trials, the rejection or non-acceptance of
any new drug application by one or more regulatory authorities and,
more generally, uncertainties related to the regulatory process
(including whether or not the regulatory authorities will accept
the Company’s conclusions regarding Macrilen™ following its
comprehensive review of the Phase 3 study data described elsewhere
in this press release), the ability of the Company to efficiently
commercialize one or more of its products or product candidates,
the degree of market acceptance once our products are approved for
commercialization, our ability to take advantage of business
opportunities in the pharmaceutical industry, our ability to
protect our intellectual property, and the potential of liability
arising from shareholder lawsuits and general changes in economic
conditions. Investors should consult the Company’s quarterly and
annual filings with the Canadian securities commissions and the SEC
for additional information on risks and uncertainties. Given these
uncertainties and risk factors, readers are cautioned not to place
undue reliance on these forward-looking statements. We disclaim any
obligation to update any such factors or to publicly announce any
revisions to any of the forward-looking statements contained herein
to reflect future results, events or developments, unless required
to do so by a governmental authority or applicable law.
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Aeterna Zentaris Inc.Philip A. Theodore, 843-900-3223Senior Vice
Presidentir@aezsinc.com
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